Improving therapeutic potential of non-viral minimized DNA vectors.
Improving therapeutic potential of non-viral minimized DNA vectors.
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DOI：
10.18609/cgti.2020.163
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发表时间：
2020-11
期刊：
影响因子：
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通讯作者：
Zechiedrich L
中科院分区：
文献类型：
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作者：
Arévalo-Soliz LM;Hardee CL;Fogg JM;Corman NR;Noorbakhsh C;Zechiedrich L
在最近的一项基于AAV的X连锁肌管性肌病临床试验中，三名患者悲惨地死亡，再次强调了对安全可靠的基因传递载体的迫切需要。非病毒最小化DNA载体为满足这一需求提供了一种可能的方法。最近使用最小化DNA载体的临床前结果在癌症治疗、干细胞治疗、干细胞再编程和其他用途方面取得了令人振奋的结果。然而，这些载体的广泛临床应用仍有待实现。媒介设计和生产方面的进一步进展正在进行中。操纵非病毒最小化DNA载体的特定形状是一项有趣而有前途的潜在发展成果。通过改善细胞摄取和生物分布特异性，这种方法可能会影响基因治疗、DNA纳米技术和个性化医学。
The tragic deaths of three patients in a recent AAV-based X-linked myotubular myopathy clinical trial highlight once again the pressing need for safe and reliable gene delivery vectors. Non-viral minimized DNA vectors offer one possible way to meet this need. Recent pre-clinical results with minimized DNA vectors have yielded promising outcomes in cancer therapy, stem cell therapy, stem cell reprograming, and other uses. Broad clinical use of these vectors, however, remains to be realized. Further advances in vector design and production are ongoing. An intriguing and promising potential development results from manipulation of the specific shape of non-viral minimized DNA vectors. By improving cellular uptake and biodistribution specificity, this approach could impact gene therapy, DNA nanotechnology, and personalized medicine.